Slide 1

Report
Clinical Trials in IPF
Dr Helen Parfrey
Are clinical trials needed ?
• Essential to determine if a drug therapy is
beneficial
• Identify who will benefit the most from the treatment
• Side effects associated with the drug therapy
• Information about the disease
How to determine if a drug treatment is
effective?
• “Controlled” - compare the drug treatment to a placebo
• “Blinded” - the study co-ordinators and patients do not know what
treatment has been given
• “Endpoints” - markers to indicate if the drug is beneficial
• IPF trials use lung function tests (FVC), hospital admissions, survival,
quality of life, measures of breathlessness
• “Powered” - sufficient number of patients
• “Inclusion and exclusion criteria” – how to select patients for a study
Have you taken part in a clinical trial for IPF?
Exciting time for IPF
But, several studies have shown that the treatments
have not been effective for IPF
• Interferon gamma studies (INSPIRE)
• Bosentan studies (BUILD series)
• Anti-TNFα studies (Etanercept)
• Tyrosine kinase inhibitor (Imatinib)
Triple therapy: IFIGENIA study
Limitations
All patients in the study taking prednisolone and
azathioprine
Not compare treatment with N-acetylcysteine alone
with placebo
Demedts et al New Engl J Med 2005
Interim safety analysis: PANTHER-IPF
• Phase III trial, double blind placebo controlled to assess effectiveness
of NAC
• Recruited 238 out of 390 patients with mild to moderate IPF
Mortality
Hospitalisation
SAE
Pred/Aza/NAC
Placebo
11%
1%
29%
8%
31%
9%
• Patient enrolment into Pred/Aza/NAC ceased
Anticoagulation therapy for IPF
Limitations
Small number of patients
Not blinded
High drop out in treatment group
No exclusion of PE as cause for deterioration
Kubo et al, Chest 2005
New treatments - Pirfenidone
Noble PW et al, Lancet 2011
Cochrane Review of Pirfenidone Studies
• First licensed treatment for mild to moderate IPF
• FVC≥50%, TLCO≥35%, 6MWD ≥150m
• Named patient programme
• NICE evaluation currently
Clinical Trial
• Tyrosine kinase inhibitor BIBF 1120
• Phase II trial, placebo controlled, 432 patients with IPF
Richeldi L et al New Engl J Med 2011
How to find out about clinical trials in IPF?
• Ask your hospital doctor
• Clinicaltrials.gov website
• Details of clinical trials in all areas of medicine
• Use search function to look for trials related to IPF
Current Clinical Trials in IPF
116 studies in IPF
22 therapeutic trials
On-going clinical trials
•
BIBF 1120
•
QAX576
•
Thalidomide
•
Sildenafil
•
Plasma exchange, rituximab and steroids for acute exacerbations
•
Sirolimus
•
Losartan
•
Phase I
•
AB0024
•
PRM-151
•
STX-100
•
CC930
•
IW001
Clinicaltrials.gov
Summary
• Importance of clinical trials in evaluating new
treatments for IPF
• Carefully designed
• Expotential increase in clinical trials for IPF
• Change how IPF is treated
Thank you for participating in the
studies

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